Supplementary tables: The cost–effectiveness, of pegcetacoplan compared with ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria, in a UK setting

These are peer-reviewed supplementary materials for the article 'The cost–effectiveness, of pegcetacoplan compared with ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria, in a UK setting' published in the Journal of Comparative Effectiveness Research.Supplementary Table 1: Estimated utilities based on the Longworth and McKenzie algorithms Supplementary Table 2: Key scenario analysesSupplementary Table 3: Probability of developing adverse events per cycle Supplementary Table 4: Iron overload disutilitySupplementary Table 5: Number of physician visits/tests per cycle1 and unit healthcare costsSupplementary Table 6: Ravulizumab iron chelation costsSupplementary Table 7: Intravascular haemolysis breakthrough cost for patients who discontinue pegcetacoplan due to intravascular haemolysis breakthroughSupplementary Table 8: Unit costs of managing adverse eventsSupplementary Table 9: OWSA results for the 10 parameters that contribute the largest difference to the NMB for pegcetacoplan versus ravulizumabAim: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder characterized by hemolytic anemia, bone marrow failure and thrombosis. We evaluated, the cost–effectiveness of pegcetacoplan, a novel proximal C3 inhibitor, versus ravulizumab in patients with PNH and hemoglobin levels Materials & methods: A Markov cohort framework model, based on the data from the pivotal trial of pegcetacoplan (PEGASUS/NCT03500549), evaluated lifetime costs and outcomes. Patients transitioned through 3 PNH hemoglobin level/red blood cell transfusion health states. Results: Pegcetacoplan provides lower lifetime costs/greater quality-adjusted life years (£6,409,166/14.694QALYs, respectively) versus ravulizumab (£6,660,676/12.942QALYs). Conclusion: Pegcetacoplan is associated with enhanced anemia control, greater QALYs and reduced healthcare costs versus ravulizumab in the UK healthcare and social services setting.